DELIVERING ON THEPROMISE OF GENE THERAPY

Creating transformative therapies for patients with rare diseases.

EXCELLENCE INGENE THERAPY

through modular technology, proprietary manufacturing and the experience to succeed.

Research & Development

Our mission at uniQure is to deliver curative gene therapies that transform the lives of patients. We have an ongoing clinical program in hemophilia B and preclinical proof-of-concept for a gene therapy in Huntington's disease.